Is busulfan a useful drug to improve successful transplantation in the treatment of acute leukemia?

This study looked at the effect of using busulfan to delete blood cancer cells from the system before replacing them with normal cells in the treatment of leukaemia. The study found that busulfan therapy was effective in removing blood cancer cells before replacement, in the treatment of high-risk acute leukaemia.

Acute leukemia is often treated by first destroying the cancerous blood cells (myeloablative conditioning), then replacing them with donor stem cell transplantation. In a haploidentical transplantation, the cells are a partial genetic match to those of the patient.

Different types of chemotherapy and radiotherapy may be used in myeloblative conditioning. Transplant rejection and relapse are a common complications. Busulfan is a form of chemotherapy. Whether this treatment can help to reduce relapse rates is under investigation.

65 patients with acute leukemia were included in the study. 25 (38%) patients received myeloablative conditioning that included busulfan for 3 days. 40 (62%) patients received busulfan for 4 days. Patients were followed for an average of 31 months.

Neutrophil and platelet engraftment (when the transplanted cells begin producing new blood cells) was effective in 97% of all patients at 30 days and 86% at 3 months. Acute graft versus host disease (GVHD – when transplanted cells attack healthy tissue) occurred in 28.6%. 6.3% of patients experienced severe GVHD. Chronic (late) GVHD occurred in 27.5% of patients.

After 30 months follow-up, 56% of patients were event free (they did not experience an event such as disease progression or death). The 30-month overall survival rate (time from treatment until death from any cause) was 54.5%.

Side effects included inflammation of the digestive tract (86%), bladder pain with blood in the urine (29%), fever (76%) and viral infection (58%).

The study concluded that myeloablative therapy with busulfan was an effective treatment to improve successful transplantation in patients with acute leukemia.

The study had a small sample size.